Public Engagement and Clinical Trials New Models and Disruptive Technologies: Workshop Summary (2012) / Chapter Skim
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7 Novel Clinical Trial Designs
7 Novel Clinical Trial Designs
Pages 55-64
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From page 55... ...
, FDA, from a practical standpoint, can we create a library of case studies so that people can see how these methods work? ADAPTIVE CLINICAL TRIAL DESIGNS1 While the annual number of new drug approvals in the United States has remained relatively flat for the past several decades -- hovering more or less between 18 to 25 -- what has not been constant are the costs of drug trials, which have been increasing at about triple the inflation rate.
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From page 56... ...
Many phase III trials that fail have problems that can be traced to phase I and II trials that did not produce the quality of information needed for the confirmatory trial to be designed appropriately. Improved clinical trial designs hold great promise for making the clinical trial enterprise more efficient, primarily by earlier detection of inadequate benefit.
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From page 57... ...
For example, "adaptive dose-finding randomization" helps the researcher decide to drop treatment arms based on initial responses, whether due to toxicity, efficacy, or both; and even in a phase III confirmatory trial, where adjustments must be made cautiously, group sequential procedures are an accepted way to gain information that can lead to midcourse corrections or even trial termination. A real-world trial conducted in LVAD recipients -- people with endstage heart failure surgically implanted with mechanical heart pumps -- provides an example.
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From page 58... ...
USING POINT-OF-CARE CLINICAL TRIALS TO CREATE A LEARNING HEALTH CARE SYSTEM3 Randomized clinical trials remain the gold standard for determining a treatment's safety and efficacy, but their high costs and extended timelines and the delayed integration of their results into clinical care are problematic. Observational studies are less expensive and produce quicker results, but their findings are less reliable.
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From page 59... ...
The VA is well positioned to try this approach for several reasons: its clinicians are interested in it; VA patients continually return to the system over a period of years; and the VA has a sophisticated EHR system, which allows patient records to be accessed from any VA facility. According to Fiore, point-of-care clinical research is a quality improvement strategy, and it has a number of advantages over traditional research methods: • he studies compare existing approved drugs with known toxici T ties and therefore do not involve IND applications or require IRB approval -- they ask "which is better?
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From page 60... ...
Administratively, point-of-care research is facilitated by what Fiore terms a "rational approach" to regulatory oversight and obtaining informed consent. The question of whether clinicians are participating in a trial, versus merely going about their regular business, is an important BOX 7-1a Point-of-Care Clinical Trial Pilot Study To test the feasibility of point-of-care research, VA investigators at the eight VA hospitals in the six-state New England region are comparing weight-based versus sliding-scale determinations of insulin dose in non-intensive care unit patients with diabetes.
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From page 61... ...
Yet, the academic infrastructure has not even begun to dismantle these silos. Additionally, Zuckerman mentioned that the training environment needs to change, so that medical schools produce physicians who are clinical trialists and clinical research courses become a standard part of the curriculum.
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From page 62... ...
The agency is working to focus the clini cal trials monitoring program on trial sites where the most problems are likely, rather than treating all sites equally. In an effort to build quality into a clinical trial from the beginning, Pfizer and FDA staff are conducting a pilot test in which they are simultaneously designing a phase III study and its monitoring program.
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From page 63... ...
Adaptive designs may be particularly helpful to device developers, as many of them are small companies with corre spondingly small research budgets. But even large device manufacturers may want trial results quickly, because a device's life cycle is often rela tively short.
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